NCATS-FDA Team Up for Rare Disease Day 2025

Since 2008, the last day of February has been recognized around the world as Rare Disease Day, a time to spotlight 300 million people living with rare diseases and the research being done to combat those diseases.

This year, NCATS has teamed up with the U.S. Food and Drug Administration (FDA) to co-sponsor Rare Disease Day 2025.The annual event aims to raise awareness about rare diseases, the people they affect and the collaborations that address challenges and advance research for new treatments.

On Feb. 27 and 28, Rare Disease Day will feature personal and scientific stories from researchers, advocates and people living with rare diseases. Topics will include the clinical and regulatory applications of artificial intelligence in rare diseases; recent approvals and successes in gene therapy; drug development through innovative trial design, and ensuring access across the rare disease treatment pipeline.

The sessions are open to staff and the public—including patients, patient advocates, caregivers, health care providers, researchers, trainees, students and industry representatives. The hybrid event will take place in person at the Natcher Conference Center and on videocast.

To learn more and to register, visit https://ncats.nih.gov/rdd.